WO2008109375A3 - Nucleic acid compounds for inhibiting pik3c gene expression and uses thereof - Google Patents

Nucleic acid compounds for inhibiting pik3c gene expression and uses thereof Download PDF

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Publication number
WO2008109375A3
WO2008109375A3 PCT/US2008/055377 US2008055377W WO2008109375A3 WO 2008109375 A3 WO2008109375 A3 WO 2008109375A3 US 2008055377 W US2008055377 W US 2008055377W WO 2008109375 A3 WO2008109375 A3 WO 2008109375A3
Authority
WO
WIPO (PCT)
Prior art keywords
pik3c
nucleic acid
gene expression
inhibiting
acid compounds
Prior art date
Application number
PCT/US2008/055377
Other languages
French (fr)
Other versions
WO2008109375A4 (en
WO2008109375A2 (en
WO2008109375A9 (en
Inventor
Steven C Quay
James Mcswiggen
Narendra K Vaish
Mohammad Ahmadian
Original Assignee
Mdrna Inc
Steven C Quay
James Mcswiggen
Narendra K Vaish
Mohammad Ahmadian
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Mdrna Inc, Steven C Quay, James Mcswiggen, Narendra K Vaish, Mohammad Ahmadian filed Critical Mdrna Inc
Publication of WO2008109375A2 publication Critical patent/WO2008109375A2/en
Publication of WO2008109375A9 publication Critical patent/WO2008109375A9/en
Publication of WO2008109375A3 publication Critical patent/WO2008109375A3/en
Publication of WO2008109375A4 publication Critical patent/WO2008109375A4/en
Priority to US12/552,082 priority Critical patent/US20100105134A1/en
Priority to US13/327,545 priority patent/US20130011922A1/en

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed

Abstract

The present disclosure provides meroduplex ribonucleic acid molecules (mdRNA) capable of decreasing or silencing PIK3C gene expression. An mdRNA of this disclosure comprises at least three strands that combine to form at least two non-overlapping double-stranded regions separated by a nick or gap wherein one strand is complementary to a PIK3C mRNA. In addition, the meroduplex may have at least one uridine substituted with a 5-methyluridine, a nucleoside replaced with a locked nucleic acid, or optionally other modifications, and any combination thereof. Also provided are methods of decreasing expression of a PIK3C gene in a cell or in a subject to treat a PIK3C-related disease.
PCT/US2008/055377 2007-03-02 2008-02-28 Nucleic acid compounds for inhibiting pik3c gene expression and uses thereof WO2008109375A2 (en)

Priority Applications (2)

Application Number Priority Date Filing Date Title
US12/552,082 US20100105134A1 (en) 2007-03-02 2009-09-01 Nucleic acid compounds for inhibiting gene expression and uses thereof
US13/327,545 US20130011922A1 (en) 2007-03-02 2011-12-15 Nucleic acid compounds for inhibiting gene expression and uses thereof

Applications Claiming Priority (12)

Application Number Priority Date Filing Date Title
US93494007P 2007-03-02 2007-03-02
US60/934,940 2007-03-02
US93493007P 2007-03-16 2007-03-16
US60/934,930 2007-03-16
US94945007P 2007-07-12 2007-07-12
US60/949,450 2007-07-12
US95117007P 2007-07-20 2007-07-20
US95116807P 2007-07-20 2007-07-20
US95116707P 2007-07-20 2007-07-20
US60/951,168 2007-07-20
US60/951,167 2007-07-20
US60/951,170 2007-07-20

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2008/055376 Continuation-In-Part WO2008109374A1 (en) 2007-03-02 2008-02-28 Nucleic acid compounds for inhibiting mapk gene expression and uses thereof

Related Child Applications (3)

Application Number Title Priority Date Filing Date
PCT/US2008/055345 Continuation-In-Part WO2008109355A1 (en) 2007-03-02 2008-02-28 Nucleic acid compounds for inhibiting srd5a2 gene expression and uses thereof
AU2009212920A Division AU2009212920A1 (en) 2007-03-02 2009-09-01 Nucleic acid compounds for inhibiting gene expression and uses thereof
US12/552,082 Continuation-In-Part US20100105134A1 (en) 2007-03-02 2009-09-01 Nucleic acid compounds for inhibiting gene expression and uses thereof

Publications (4)

Publication Number Publication Date
WO2008109375A2 WO2008109375A2 (en) 2008-09-12
WO2008109375A9 WO2008109375A9 (en) 2008-12-11
WO2008109375A3 true WO2008109375A3 (en) 2009-03-05
WO2008109375A4 WO2008109375A4 (en) 2009-04-30

Family

ID=39639303

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2008/055377 WO2008109375A2 (en) 2007-03-02 2008-02-28 Nucleic acid compounds for inhibiting pik3c gene expression and uses thereof

Country Status (1)

Country Link
WO (1) WO2008109375A2 (en)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2009152346A2 (en) * 2008-06-11 2009-12-17 Intradigm Corporation Compositions comprising phosphoinositide 3-kinase sirna and methods of use thereof
ES2585829T3 (en) * 2009-12-29 2016-10-10 Curna, Inc. Treatment of diseases related to tumor protein 63 (p63) by inhibition of natural antisense transcription to p63
US9453261B2 (en) * 2011-09-20 2016-09-27 The George Washington University Alternative splicing variants of genes associated with prostate cancer risk and survival
US10226424B2 (en) * 2012-08-12 2019-03-12 Mylisa Parette Therapeutic calcium phosphate nanoparticle incorporating siRNA useful in treating disease

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2003070918A2 (en) * 2002-02-20 2003-08-28 Ribozyme Pharmaceuticals, Incorporated Rna interference by modified short interfering nucleic acid
WO2005091849A2 (en) * 2004-03-02 2005-10-06 The Johns Hopkins University Mutations of the pik3ca gene in human cancers
WO2007056153A2 (en) * 2005-11-04 2007-05-18 Nastech Pharmaceutical Company Inc. Peptide-dicer substrate rna conjugates as delivery vehicles for sirna
WO2007107162A2 (en) * 2006-03-23 2007-09-27 Santaris Pharma A/S Small internally segmented interfering rna

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2003070918A2 (en) * 2002-02-20 2003-08-28 Ribozyme Pharmaceuticals, Incorporated Rna interference by modified short interfering nucleic acid
WO2005091849A2 (en) * 2004-03-02 2005-10-06 The Johns Hopkins University Mutations of the pik3ca gene in human cancers
WO2007056153A2 (en) * 2005-11-04 2007-05-18 Nastech Pharmaceutical Company Inc. Peptide-dicer substrate rna conjugates as delivery vehicles for sirna
WO2007107162A2 (en) * 2006-03-23 2007-09-27 Santaris Pharma A/S Small internally segmented interfering rna

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
BRAMSEN J B ET AL: "Improved silencing properties using small internally segmented interfering RNAs", NUCLEIC ACIDS RESEARCH, OXFORD UNIVERSITY PRESS, SURREY, GB, vol. 35, no. 17, 28 July 2007 (2007-07-28), pages 5886 - 5897, XP002467844, ISSN: 0305-1048 *
LEUSCHNER P J F ET AL: "Cleavage of the siRNA passenger strand during RISC assembly in human cells", EMBO REPORTS, XX, XX, vol. 7, no. 3, 1 March 2006 (2006-03-01), pages 314 - 320, XP002467845 *
MATRANGA CHRISTIAN ET AL: "Passenger-strand cleavage facilitates assembly of siRNA into Ago2-containing RNAi enzyme complexes", CELL,, vol. 123, no. 4, 1 November 2005 (2005-11-01), pages 607 - 620, XP002484663 *
MILLS JANINE B ET AL: "Origin of the intrinsic rigidity of DNA", NUCLEIC ACIDS RESEARCH,, vol. 32, no. 13, 1 January 2004 (2004-01-01), pages 4055 - 4059, XP002484664 *

Also Published As

Publication number Publication date
WO2008109375A4 (en) 2009-04-30
WO2008109375A2 (en) 2008-09-12
WO2008109375A9 (en) 2008-12-11

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